The psychometric properties of the EQ-5D-Y have not been widely tested in severely ill children. The aim of this study was to assess and compare the validity and responsiveness of the EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric inpatients with haematological malignancies and caregivers. Respondents completed the interviewer-administered self-complete or proxy version of the EQ-5D-Y-3L and EQ-5D-Y-5L and an overall health assessment twice on different days. Known-groups validity was assessed by comparing patients who differed in overall health and Eastern Cooperative Oncology Group (ECOG) performance. Responsiveness to worsened health was assessed using standardised effect size (SES) for patients with worsened ECOG grade, self-reported rating, or chemotherapy initiation. Ninety-six dyads completed the baseline questionnaires. A smaller proportion of patients reported “no problems” on the EQ-5D-Y-5L compared to EQ-5D-Y-3L for most of the five dimensions. Patients in poor health reported more problems in all dimensions and had higher EQ-5D-Y-5L level sum score, lower EQ VAS and EQ-5D-Y-3L index scores (Cohen’s d ES: 0.32–1.38 for patients; 0.50–2.05 for caregivers). There was a mild to good responsiveness to worsened health condition based on ECOG (SES: 0.14–0.61 for patients; 0.40–0.96 for caregivers), suggesting the proxy version was slightly responsive than the self-complete version of both instruments. The results demonstrated validity and responsiveness for both the self-complete and proxy versions of the EQ-5D-Y-3L and EQ-5D-Y-5L. The proxy and 5-level versions of the instrument were more sensitive than the self-complete and 3-level versions in this patient group.

EQ-5D-Y 的心理测量特性尚未在重症儿童中进行广泛测试。本研究的目的是评估和比较 EQ-5D-Y-3L 和 EQ-5D-Y-5L 在血液系统恶性肿瘤儿科住院患者和护理人员中的有效性和反应性。受访者在不同日期完成两次由访谈员管理的自我完成或代理版本的 EQ-5D-Y-3L 和 EQ-5D-Y-5L 以及整体健康评估。通过比较总体健康状况和东部肿瘤合作组 (ECOG) 表现不同的患者来评估已知组的有效性。对于 ECOG 分级恶化、自我报告评级或开始化疗的患者，使用标准化效应量 (SES) 评估对健康状况恶化的反应。九十六人完成了基线调查问卷。与 EQ-5D-Y-3L 相比，在 EQ-5D-Y-5L 上报告五个维度的大部分“没有问题”的患者比例较小。健康状况不佳的患者在各维度上报告的问题较多，EQ-5D-Y-5L 水平总分较高，EQ VAS 和 EQ-5D-Y-3L 指数得分较低（Cohen's d ES：患者 0.32–1.38；0.50–照顾者为 2.05）。根据 ECOG，对恶化的健康状况有轻微到良好的反应（SES：患者为 0.14-0.61；护理人员为 0.40-0.96），这表明代理版本比两种工具的自我完成版本的反应稍弱。结果证明了 EQ-5D-Y-3L 和 EQ-5D-Y-5L 的自我完成版本和代理版本的有效性和响应能力。在该患者组中，仪器的代理版本和 5 级版本比自我完成版本和 3 级版本更敏感。